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Use of HSC- Bone Marrow Transplant (BMT)

Importance of HSC

Stem cell transplantation is now used globally in the treatment of many malignant and non-malignant haematologic conditions. With the progress of medical science, new researches and clinical trials, the benefits of transplant have been extended to maximum number of patients and elderly people as well.

Bone marrow transplants are performed when a person’s marrow is not healthy enough to function normally because of chronic infections, diseases or cancer treatments.

There are certain diseases such as Leukaemia (malignant) and Thalassemia (non-malignant) which are treated with traditional treatment of regular blood transfusion or chemotherapy. However Bone Marrow/Stem cell transplant is the only curative treatment option available. People experiencing bone marrow (blood cell) failure disorders also need HSC transplant.

Stem cell transplantation has become most important remedial option for a number of non-malignant and malignant disorders disturbing the bone marrow resulting serious health issues. Autoimmune diseases, haemoglobinopathies, immune-deficiencies and other cancerous disease like leukaemia, lymphoma and myeloma.

For many of these disorders, early diagnosis is crucial in achieving the desired results as transplantation becomes difficult when significant end-organ damage sets in. Major unsolved problems, including toxicity of conditioning regimens, graft-versus-host disease, and donor availability, need to be addressed.

HSC–bone marrow transplant has proved to be beneficial in many diseases, following are few of them:

Non-malignant Conditions

1) Aplastic Anemia

This is an autoimmune disease that causes white blood cells to attack blood stem cells, resulting in low red cell, white cell and platelet counts. This is rare and serious disorder which can develop at any age. It may occur suddenly or gradually and turn out to be worse with time. Aplastic anemia causes weakness, uncontrolled bleeding and makes a person prone to infections.

Treatment for aplastic anemia may include medications, blood transfusions or a stem cell transplant.

2) Thalassemia Major

This is an inherited blood disorder where the body makes an abnormal form of haemoglobin, an integral part of red blood cells. People with this condition produce either no or too little haemoglobin. Haemoglobin is the protein in red blood cells that carries oxygen. The disorder results in large numbers of red blood cells being destroyed, which leads to anemia..Cord blood stem cell transplant is an effective treatment for Thalassemia

3) Sickle Cell Anemia

Cells in tissues need a steady supply of oxygen to work well. In a healthy person’s body, hemoglobin in red blood cells work as transporter and carries oxygen in the lungs and also to all the tissues of the body. People with SCD have abnormal hemoglobin, called hemoglobin S. or sickle hemoglobin, in their red blood cells.

Normal red blood cells are in round shape but when there is a condition where these cells grow in sickle-shaped (long and twisted like the farming tool called sickle) instead of round shape of a healthy cell, it is called sickle cell anaemia which is a genetic blood disorder.

Since sickle-shaped cells are twisted and non-flexible, they stick to vessel walls, causing a blockage that slows down or completely stop the blood flow. In this situation, oxygen can’t reach to nearby tissues.

These abnormal cells die very soon as they are unable to sustain due to their abnormal non-performing shape. Hence shortage of healthy red blood cells can result into poor oxygen delivery in the body hampering blood flow causing pain (sickle cell crisis). Sickle Cell Disease can harm a person’s spleen, brain, eyes, lungs, liver, heart, kidneys, penis, joints, bones, or skin. This can also lead to organ damage.

Treatments for sickle cell anemia include medications, blood transfusions and a bone-marrow transplant. At the present time, hematopoietic stem cell transplantation (HSCT) is the only cure for SCD.

4) Hemophagocytic Syndrome

Hemophagocytic lymphohistiocytosis  (HLH) is a condition in which the body makes too many activated immune cells (macrophages and lymphocytes). It is a life-threatening immunodeficiency which affects people of all ages.

Early recognition is crucial and without prompt treatment HLH is often fatal. Treatment includes targeting the underlying disorder and controlling the immune dysregulation. Etoposide, dexamethasone, and ultimately HSCT are the mainstay for the majority of patients with HLH.

Bone-marrow transplant is one of the best treatment options available for this disorder.



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